A new gene therapy treatment for Duchenne muscular dystrophy shows promise of not only arresting the decline of the muscles of those affected by this inherited genetic disease, but perhaps, in the future, repairing those muscles.
University of Washington School of Medicine /UW Medicine Jul 18 2024 A new gene therapy treatment for Duchenne muscular dystrophy shows promise of not only arresting the decline of the muscles of those affected by this inherited genetic disease, but perhaps, in the future, repairing those muscles.
Currently, there is no cure for the disease and available treatments and drugs only slow down the disease. In the case of Duchenne muscular dystrophy – the topic of this study – patients, all males because the gene is on the X chromosome, begin exhibiting symptoms around age four and usually die their 20s or 30s. UW Medicine neurologist and geneticist, Dr.
The new method, which has had success on mouse models, uses a series of adeno-associated viral vectors or AAVs, which are tiny shuttles derived from a virus that are being used to deliver gene therapies into human cells. Instead of one AAV, this gene therapy uses a series of AAVs which take parts of the therapeutic protein inside the muscles, along with embedded instructions to begin assembling the necessary genetic fix once within the body.
Related StoriesIn the lab, this method has not only halted further progression of the disease, but it has been able to reverse much of the pathology associated with dystrophy. Eventually, Chamberlain and Tasfaout hope that this method might lead to a reversal of the muscle wasting and restore normal health of the muscle tissue.
Gene Therapy Muscular Dystrophy Drugs Duchenne Muscular Dystrophy Dystrophin Genetic Laboratory Medicine Muscle Protein Research
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